A gene therapy trial cured children of a disease that prevented them from walking and talking, the patients are now in school.
The therapy corrected errors in the children's DNA responsible for the debilitating disease, the BBC reported. Similar therapy was used to cure a genetic disorder affecting the immune system.
One of the conditions, called metachromatic leukodystrophy , doesn't show up until the baby is about one or two years old. The child's brain health begins to slowly deteriorate around that age.
The other genetically curable illness, Wiskott-Aldrich syndrome, compromises the immune system leading to frequent infections and even cancers. It can also cause the immune system to attack parts of the body.
A team of Italian scientists fixed the harmful gene mutations with a genetically modified virus. It is injected into the mutated bone marrow cells in order to "infect" them with corrected genetic information.
The study chose three children who had a strong family history of metachromatic leukodystrophy to participate in the trial. The treatments were administered before the infants started showing signs of the condition.
"The outcome has been very positive, they're all in very good condition, with a normal life and going to kindergarten at an age when their siblings were unable to talk," Dr. Alessandra Biffi said. "It is something which is very pleasing to us."
The treatments were not without side effects, and cannot be used on a larger scale until the trial participants are followed for a longer period of time, however the repercussions weren't serious enough to deter the researchers.
"The therapy is not only safe, but also effective and able to change the clinical history of these severe diseases," Prof Luigi Naldini, of the San Raffaele Telethon Institute for Gene Therapy said. "After 15 years of effort and our successes in the laboratory, but frustration as well, it's really exciting to be able to give a concrete solution to the first patients."
The team hopes the new findings will provide a widespread cure for the tested diseases, and will lead to the cure of other conditions as well.
